Mirum Pharmaceuticals announced Monday that their investigational medication volixibat successfully achieved its primary objective in a clinical trial focused on alleviating intense itching experienced by patients suffering from a rare liver condition.
The research involved 158 participants who received either volixibat at a dosage of 20 mg twice per day or a placebo treatment. Researchers focused their primary analysis on 111 patients experiencing moderate to severe itching symptoms, while an additional 47 participants with mild symptoms were evaluated in a separate analysis.
Primary sclerosing cholangitis, commonly referred to as PSC, is a condition that triggers inflammation and creates scar tissue in the bile ducts, causing them to narrow. This process results in bile accumulation, damage to the liver, and can progress to complete liver failure.
Among the primary study group, participants taking volixibat experienced a reduction in itching severity scores of 2.72 points compared to their baseline measurements, while those receiving placebo saw only a 1.08-point decrease. This created a statistically meaningful difference of 1.64 points favoring the experimental treatment, according to Mirum’s findings.
The pharmaceutical company stated that these outcomes demonstrate volixibat’s potential to become the initial approved therapy specifically targeting cholestatic pruritus in PSC patients.
“The takeaway here is very straightforward – trial is clearly positive & data is in line with expectations,” commented Evercore ISI analyst Gavin Clark-Gartner.
Mirum has arranged a preliminary meeting with the U.S. Food and Drug Administration for summer 2026 and intends to submit their application for regulatory approval during the latter half of that year.
